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# Rare Disease Drug Development Guidance
- URL: https://www.fdaweb.com/rare-disease-drug-development-guidance/
- Published: 2024-01-02T12:00:00.000Z
- Updated: 2026-09-14T14:19:16.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5156024

FDA has published a guidance, *Rare Diseases: Considerations for the Development of Drugs and Biological Products*, to assist sponsors in conducting efficient and successful development programs for drugs to treat rare diseases. The [document](https://www.fda.gov/regulatory-information/search-fda-guidance-documents/rare-diseases-considerations-development-drugs-and-biological-products?ref=fdaweb.com) notes that while the statutory requirements for marketing approval for drugs to treat rare and common diseases are the same and have the same issues as in other drug development programs, the issues are frequently more difficult to address in the context of rare diseases for which there is often limited medical and scientific knowledge, poorly understood natural history data, sample size constraints, and lack of drug development experience.

The guidance does not discuss the general issues of statistical analysis, which are addressed in other guidances.